Avidity Biosciences, which received early funding from CureDuchenne, plans to submit for US FDA Accelerated Approval at the end of 2025 for Delpacibart zotadirsen (del-zota), an exon-skipping experimental therapeutic for […]
CureDuchenne is delighted to share the positive updates from REGENXBIO on their Phase 1/2 open-label clinical trial of RGX-202, an AAV8-delivered microdystrophin with an extended C-terminal domain. RGX-202 demonstrated robust […]
Avidity Biosciences, which received early funding from CureDuchenne, announced positive initial data from their EXPLORE44 Trial for individuals with Duchenne amenable to skipping exon 44. Their experimental exon-skipping agent, AOC […]
We at CureDuchenne are excited to acknowledge the recent announcement by the United States Food and Drug Administration (FDA) about the creation of the “Rare Disease Innovation Hub.” This new […]
REGENXBIO announced they are expanding their AFFINITY DUCHENNE Trial, which delivers a micro-dystrophin via AAV8, to include a new cohort of younger patients. The trial, which has been enrolling ambulatory […]
PepGen Announces First Patient Dosed in CONNECT1-EDO51 Phase 2 Clinical Trial of PGN-EDO51 for Duchenne Muscular Dystrophy Patients Amenable to Exon 51 Skipping
Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients
As an early funder of Avidity Biosciences, we are pleased to share that AOC 1044, designed to skip exon 44 in the dystrophin gene, produced significant exon skipping in healthy volunteers.
– First participant is expected to be dosed in September 2023 with data anticipated in the second half of 2024 – – Cash runway extended through the end of 2025 […]
